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Volumn 17, Issue 1 SUPPL, 2011, Pages

Chronic Granulomatous Disease: Lessons from a Rare Disorder

Author keywords

Chronic granulomatous disease; Gene therapy; Hematopoietic cell transplantation; NADPH oxidase

Indexed keywords

ALEMTUZUMAB; BUSULFAN; COTRIMOXAZOLE; CYCLOPHOSPHAMIDE; DICLOXACILLIN; FLUDARABINE; ITRACONAZOLE; LENTIVIRUS VECTOR; MELPHALAN; POSACONAZOLE; RAPAMYCIN; RECOMBINANT GAMMA INTERFERON; THYMOCYTE ANTIBODY; TREOSULFAN; VORICONAZOLE;

EID: 78650647287     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2010.09.008     Document Type: Article
Times cited : (112)

References (45)
  • 1
    • 0034040532 scopus 로고    scopus 로고
    • Chronic granulomatous disease: report on a national registry of 368 patients
    • Winkelstein J.A., Marino M.C., Johnston R.B., et al. Chronic granulomatous disease: report on a national registry of 368 patients. Medicine (Baltimore) 2000, 79:155-169.
    • (2000) Medicine (Baltimore) , vol.79 , pp. 155-169
    • Winkelstein, J.A.1    Marino, M.C.2    Johnston, R.B.3
  • 2
    • 0037149510 scopus 로고    scopus 로고
    • Killing activity of neutrophils is mediated through activation of proteases by K+ flux
    • Reeves E.P., Lu H., Jacobs H.L., et al. Killing activity of neutrophils is mediated through activation of proteases by K+ flux. Nature 2002, 416:291-297.
    • (2002) Nature , vol.416 , pp. 291-297
    • Reeves, E.P.1    Lu, H.2    Jacobs, H.L.3
  • 3
    • 1542287347 scopus 로고    scopus 로고
    • Neutrophil extracellular traps kill bacteria
    • Brinkmann V., Reichard U., Goosmann C., et al. Neutrophil extracellular traps kill bacteria. Science 2004, 303:1532-1535.
    • (2004) Science , vol.303 , pp. 1532-1535
    • Brinkmann, V.1    Reichard, U.2    Goosmann, C.3
  • 4
    • 70350004580 scopus 로고    scopus 로고
    • Restoration of NET formation by gene therapy in CGD controls aspergillosis
    • Bianchi M., Hakkim A., Brinkmann V., et al. Restoration of NET formation by gene therapy in CGD controls aspergillosis. Blood 2009, 114:2619-2622.
    • (2009) Blood , vol.114 , pp. 2619-2622
    • Bianchi, M.1    Hakkim, A.2    Brinkmann, V.3
  • 5
    • 33846432787 scopus 로고    scopus 로고
    • Novel cell death program leads to neutrophil extracellular traps
    • Fuchs T.A., Abed U., Goosmann C., et al. Novel cell death program leads to neutrophil extracellular traps. J Cell Biol 2007, 176:231-241.
    • (2007) J Cell Biol , vol.176 , pp. 231-241
    • Fuchs, T.A.1    Abed, U.2    Goosmann, C.3
  • 6
    • 65349179624 scopus 로고    scopus 로고
    • Chronic granulomatous disease: the European experience
    • van den Berg J.M., van Koppen E., Ahlin A., et al. Chronic granulomatous disease: the European experience. PLoS ONE 2009, 4:e5234.
    • (2009) PLoS ONE , vol.4
    • van den Berg, J.M.1    van Koppen, E.2    Ahlin, A.3
  • 7
    • 3442877820 scopus 로고    scopus 로고
    • Gastrointestinal involvement in chronic granulomatous disease
    • Marciano B.E., Rosenzweig S.D., Kleiner D.E., et al. Gastrointestinal involvement in chronic granulomatous disease. Pediatrics 2004, 114:462-468.
    • (2004) Pediatrics , vol.114 , pp. 462-468
    • Marciano, B.E.1    Rosenzweig, S.D.2    Kleiner, D.E.3
  • 8
    • 34548508186 scopus 로고    scopus 로고
    • Fulminant mulch pneumonitis: an emergency presentation of chronic granulomatous disease
    • Siddiqui S., Anderson V.L., Hilligoss D.M., et al. Fulminant mulch pneumonitis: an emergency presentation of chronic granulomatous disease. Clin Infect Dis 2007, 45:673-681.
    • (2007) Clin Infect Dis , vol.45 , pp. 673-681
    • Siddiqui, S.1    Anderson, V.L.2    Hilligoss, D.M.3
  • 9
    • 0031041884 scopus 로고    scopus 로고
    • Absence of respiratory burst in X-linked chronic granulomatous disease mice leads to abnormalities in both host defense and inflammatory response to Aspergillus fumigatus
    • Morgenstern D.E., Gifford M.A., Li L.L., Doerschuk C.M., Dinauer M.C. Absence of respiratory burst in X-linked chronic granulomatous disease mice leads to abnormalities in both host defense and inflammatory response to Aspergillus fumigatus. J Exp Med 1997, 185:207-218.
    • (1997) J Exp Med , vol.185 , pp. 207-218
    • Morgenstern, D.E.1    Gifford, M.A.2    Li, L.L.3    Doerschuk, C.M.4    Dinauer, M.C.5
  • 10
    • 38049115134 scopus 로고    scopus 로고
    • Defective tryptophan catabolism underlies inflammation in mouse chronic granulomatous disease
    • Romani L., Fallarino F., De Luca A., et al. Defective tryptophan catabolism underlies inflammation in mouse chronic granulomatous disease. Nature 2008, 451:211-215.
    • (2008) Nature , vol.451 , pp. 211-215
    • Romani, L.1    Fallarino, F.2    De Luca, A.3
  • 11
    • 77956612015 scopus 로고    scopus 로고
    • NADPH oxidase limits innate immune responses in the lungs in mice
    • Segal B.H., Han W., Bushey J.J., et al. NADPH oxidase limits innate immune responses in the lungs in mice. PLoS ONE 2010, 5:e9631.
    • (2010) PLoS ONE , vol.5
    • Segal, B.H.1    Han, W.2    Bushey, J.J.3
  • 12
    • 0038242951 scopus 로고    scopus 로고
    • Itraconazole to prevent fungal infections in chronic granulomatous disease
    • Gallin J.I., Alling D.W., Malech H.L., et al. Itraconazole to prevent fungal infections in chronic granulomatous disease. N Engl J Med 2003, 348:2416-2422.
    • (2003) N Engl J Med , vol.348 , pp. 2416-2422
    • Gallin, J.I.1    Alling, D.W.2    Malech, H.L.3
  • 13
    • 0026090433 scopus 로고
    • A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease
    • The International Chronic Granulomatous Disease Cooperative Study Group
    • A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease. N Engl J Med 1991, 324:509-516. The International Chronic Granulomatous Disease Cooperative Study Group.
    • (1991) N Engl J Med , vol.324 , pp. 509-516
  • 14
    • 0031706467 scopus 로고    scopus 로고
    • Aspergillus nidulans infection in chronic granulomatous disease
    • Segal B.H., DeCarlo E.S., Kwon-Chung K.J., et al. Aspergillus nidulans infection in chronic granulomatous disease. Medicine (Baltimore) 1998, 77:345-354.
    • (1998) Medicine (Baltimore) , vol.77 , pp. 345-354
    • Segal, B.H.1    DeCarlo, E.S.2    Kwon-Chung, K.J.3
  • 15
    • 38349105032 scopus 로고    scopus 로고
    • Modern management of chronic granulomatous disease
    • Seger R.A. Modern management of chronic granulomatous disease. Br J Haematol 2008, 140:255-266.
    • (2008) Br J Haematol , vol.140 , pp. 255-266
    • Seger, R.A.1
  • 16
    • 0037114622 scopus 로고    scopus 로고
    • Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000
    • Seger R.A., Gungor T., Belohradsky B.H., et al. Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000. Blood 2002, 100:4344-4350.
    • (2002) Blood , vol.100 , pp. 4344-4350
    • Seger, R.A.1    Gungor, T.2    Belohradsky, B.H.3
  • 17
    • 61949241213 scopus 로고    scopus 로고
    • Unrelated donor and HLA-identical sibling haematopoietic stem cell transplantation cure chronic granulomatous disease with good long-term outcome and growth
    • Soncini E., Slatter M.A., Jones L.B., et al. Unrelated donor and HLA-identical sibling haematopoietic stem cell transplantation cure chronic granulomatous disease with good long-term outcome and growth. Br J Haematol 2009, 145:73-83.
    • (2009) Br J Haematol , vol.145 , pp. 73-83
    • Soncini, E.1    Slatter, M.A.2    Jones, L.B.3
  • 18
    • 20544436474 scopus 로고    scopus 로고
    • Successful low toxicity hematopoietic stem cell transplantation for high-risk adult chronic granulomatous disease patients
    • Güngör T., Halter J., Klink A., et al. Successful low toxicity hematopoietic stem cell transplantation for high-risk adult chronic granulomatous disease patients. Transplantation 2005, 79:1596-1606.
    • (2005) Transplantation , vol.79 , pp. 1596-1606
    • Güngör, T.1    Halter, J.2    Klink, A.3
  • 19
    • 74849129102 scopus 로고    scopus 로고
    • Successful busulphan-based reduced intensity conditioning in high-risk paediatric and adult chronic granulomatous disease-The Swiss experience
    • Güngör T., Halter J., Stussi G., Scherer F., Schanz U., Seger R. Successful busulphan-based reduced intensity conditioning in high-risk paediatric and adult chronic granulomatous disease-The Swiss experience. Bone Marrow Transplant 2009, 43(Suppl 1):S75.
    • (2009) Bone Marrow Transplant , vol.43 , Issue.SUPPL 1
    • Güngör, T.1    Halter, J.2    Stussi, G.3    Scherer, F.4    Schanz, U.5    Seger, R.6
  • 20
    • 33846678722 scopus 로고    scopus 로고
    • Treatment of McLeod phenotype chronic granulomatous disease with reduced-intensity conditioning and unrelated-donor umbilical cord blood transplantation
    • Suzuki N., Hatakeyama N., Yamamoto M., et al. Treatment of McLeod phenotype chronic granulomatous disease with reduced-intensity conditioning and unrelated-donor umbilical cord blood transplantation. Int J Hematol 2007, 85:70-72.
    • (2007) Int J Hematol , vol.85 , pp. 70-72
    • Suzuki, N.1    Hatakeyama, N.2    Yamamoto, M.3
  • 21
    • 11244284216 scopus 로고    scopus 로고
    • Improved survival after unrelated donor bone marrow transplant in children with primary immunodeficiency using a reduced intensity conditioning regimen
    • Rao K., Amrolia P.J., Jones A., et al. Improved survival after unrelated donor bone marrow transplant in children with primary immunodeficiency using a reduced intensity conditioning regimen. Blood 2005, 105:879-885.
    • (2005) Blood , vol.105 , pp. 879-885
    • Rao, K.1    Amrolia, P.J.2    Jones, A.3
  • 22
    • 47249112625 scopus 로고    scopus 로고
    • Treosulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities
    • Greystoke B., Bonanomi S., Carr T.F., et al. Treosulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities. Br J Haematol 2008, 142:257-262.
    • (2008) Br J Haematol , vol.142 , pp. 257-262
    • Greystoke, B.1    Bonanomi, S.2    Carr, T.F.3
  • 23
    • 0035932520 scopus 로고    scopus 로고
    • Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft
    • Horwitz M.E., Barrett A.J., Brown M.R., et al. Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med 2001, 344:881-888.
    • (2001) N Engl J Med , vol.344 , pp. 881-888
    • Horwitz, M.E.1    Barrett, A.J.2    Brown, M.R.3
  • 24
    • 33746903944 scopus 로고    scopus 로고
    • Belgian loophole allows Swiss parents a " saviour" baby
    • Duke K. Belgian loophole allows Swiss parents a " saviour" baby. Lancet 2006, 368:355-356.
    • (2006) Lancet , vol.368 , pp. 355-356
    • Duke, K.1
  • 25
    • 12644293807 scopus 로고    scopus 로고
    • Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease
    • Malech H.L., Maples P.B., Whiting-Theobald N., et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci U S A 1997, 94:12133-12138.
    • (1997) Proc Natl Acad Sci U S A , vol.94 , pp. 12133-12138
    • Malech, H.L.1    Maples, P.B.2    Whiting-Theobald, N.3
  • 26
    • 0030045762 scopus 로고    scopus 로고
    • Genotype-dependent variability in flow cytometric evaluation of reduced nicotinamide adenine dinucleotide phosphate oxidase function in patients with chronic granulomatous disease
    • Vowells S.J., Fleisher T.A., Sekhsaria S., Alling D.W., Maguire T.E., Malech H.L. Genotype-dependent variability in flow cytometric evaluation of reduced nicotinamide adenine dinucleotide phosphate oxidase function in patients with chronic granulomatous disease. J Pediatr 1996, 128:104-107.
    • (1996) J Pediatr , vol.128 , pp. 104-107
    • Vowells, S.J.1    Fleisher, T.A.2    Sekhsaria, S.3    Alling, D.W.4    Maguire, T.E.5    Malech, H.L.6
  • 27
    • 0033656282 scopus 로고    scopus 로고
    • Use of serum-free medium with fibronectin fragment enhanced transduction in a system of gas permeable plastic containers to achieve high levels of retrovirus transduction at clinical scale
    • Malech H.L. Use of serum-free medium with fibronectin fragment enhanced transduction in a system of gas permeable plastic containers to achieve high levels of retrovirus transduction at clinical scale. Stem Cells 2000, 18:155-156.
    • (2000) Stem Cells , vol.18 , pp. 155-156
    • Malech, H.L.1
  • 28
    • 9144271097 scopus 로고    scopus 로고
    • Progress toward effective gene therapy for chronic granulomatous disease
    • Malech H.L., Choi U., Brenner S. Progress toward effective gene therapy for chronic granulomatous disease. Jpn J Infect Dis 2004, 57:S27-S28.
    • (2004) Jpn J Infect Dis , vol.57
    • Malech, H.L.1    Choi, U.2    Brenner, S.3
  • 29
    • 0037129435 scopus 로고    scopus 로고
    • Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
    • Hacein-Bey-Abina S., Le Deist F., Carlier F., et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002, 346:1185-1193.
    • (2002) N Engl J Med , vol.346 , pp. 1185-1193
    • Hacein-Bey-Abina, S.1    Le Deist, F.2    Carlier, F.3
  • 30
    • 19944421384 scopus 로고    scopus 로고
    • Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
    • Gaspar H.B., Parsley K.L., Howe S., et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004, 364:2181-2187.
    • (2004) Lancet , vol.364 , pp. 2181-2187
    • Gaspar, H.B.1    Parsley, K.L.2    Howe, S.3
  • 31
    • 51349090473 scopus 로고    scopus 로고
    • Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    • Hacein-Bey-Abina S., Garrigue A., Wang G.P., et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 2008, 118:3132-3142.
    • (2008) J Clin Invest , vol.118 , pp. 3132-3142
    • Hacein-Bey-Abina, S.1    Garrigue, A.2    Wang, G.P.3
  • 32
    • 51349158298 scopus 로고    scopus 로고
    • Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
    • Howe S.J., Mansour M.R., Schwarzwaelder K., et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 2008, 118:3143-3150.
    • (2008) J Clin Invest , vol.118 , pp. 3143-3150
    • Howe, S.J.1    Mansour, M.R.2    Schwarzwaelder, K.3
  • 33
    • 0028807727 scopus 로고
    • T lymphocyte-directed gene therapy for ADA-SCID initial trial results after 4 years
    • Blaese R.M., Culver K.W., Miller A.D., et al. T lymphocyte-directed gene therapy for ADA-SCID initial trial results after 4 years. Science 1995, 270:475-480.
    • (1995) Science , vol.270 , pp. 475-480
    • Blaese, R.M.1    Culver, K.W.2    Miller, A.D.3
  • 34
    • 0028789792 scopus 로고
    • Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients
    • Bordignon C., Notarangelo L.D., Nobili N., et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 1995, 270:470-475.
    • (1995) Science , vol.270 , pp. 470-475
    • Bordignon, C.1    Notarangelo, L.D.2    Nobili, N.3
  • 35
    • 0037189401 scopus 로고    scopus 로고
    • Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
    • Aiuti A., Slavin S., Aker M., et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002, 296:2410-2413.
    • (2002) Science , vol.296 , pp. 2410-2413
    • Aiuti, A.1    Slavin, S.2    Aker, M.3
  • 36
    • 59449098985 scopus 로고    scopus 로고
    • Gene therapy for immunodeficiency due to adenosine deaminase deficiency
    • Aiuti A., Cattaneo F., Galimberti S., et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009, 360:447-458.
    • (2009) N Engl J Med , vol.360 , pp. 447-458
    • Aiuti, A.1    Cattaneo, F.2    Galimberti, S.3
  • 37
    • 33748413936 scopus 로고    scopus 로고
    • Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning
    • Gaspar H.B., Bjorkegren E., Parsley K., et al. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther 2006, 14:505-513.
    • (2006) Mol Ther , vol.14 , pp. 505-513
    • Gaspar, H.B.1    Bjorkegren, E.2    Parsley, K.3
  • 38
    • 33645734405 scopus 로고    scopus 로고
    • Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
    • Ott M.G., Schmidt M., Schwarzwaelder K., et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006, 12:401-409.
    • (2006) Nat Med , vol.12 , pp. 401-409
    • Ott, M.G.1    Schmidt, M.2    Schwarzwaelder, K.3
  • 39
    • 76249131912 scopus 로고    scopus 로고
    • Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
    • Stein S., Ott M.G., Schultze-Strasser S., et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 2010, 16:198-204.
    • (2010) Nat Med , vol.16 , pp. 198-204
    • Stein, S.1    Ott, M.G.2    Schultze-Strasser, S.3
  • 40
    • 77949327872 scopus 로고    scopus 로고
    • Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils
    • Kang E.M., Choi U., Theobald N., et al. Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils. Blood 2010, 115:783-791.
    • (2010) Blood , vol.115 , pp. 783-791
    • Kang, E.M.1    Choi, U.2    Theobald, N.3
  • 41
    • 65249101260 scopus 로고    scopus 로고
    • The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy
    • Montini E., Cesana D., Schmidt M., et al. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest 2009, 119:964-975.
    • (2009) J Clin Invest , vol.119 , pp. 964-975
    • Montini, E.1    Cesana, D.2    Schmidt, M.3
  • 42
    • 70449427834 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy
    • Cartier N., Hacein-Bey-Abina S., Bartholomae C.C., et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009, 326:818-823.
    • (2009) Science , vol.326 , pp. 818-823
    • Cartier, N.1    Hacein-Bey-Abina, S.2    Bartholomae, C.C.3
  • 43
    • 58149469932 scopus 로고    scopus 로고
    • Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells
    • Moreno-Carranza B., Gentsch M., Stein S., et al. Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther 2009, 16:111-118.
    • (2009) Gene Ther , vol.16 , pp. 111-118
    • Moreno-Carranza, B.1    Gentsch, M.2    Stein, S.3
  • 44
    • 77956502744 scopus 로고    scopus 로고
    • A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells
    • Zhou S., Mody D., DeRavin S.S., et al. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood 2010, 116:900-908.
    • (2010) Blood , vol.116 , pp. 900-908
    • Zhou, S.1    Mody, D.2    DeRavin, S.S.3
  • 45
    • 67149117119 scopus 로고    scopus 로고
    • Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection
    • Throm R.E., Ouma A.A., Zhou S., et al. Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection. Blood 2009, 113:5104-5110.
    • (2009) Blood , vol.113 , pp. 5104-5110
    • Throm, R.E.1    Ouma, A.A.2    Zhou, S.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.